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Immunology

Antibodies to Spinal Muscular Atrophy Gene Therapy Vector May Dissipate, Allowing More Children to Receive Treatment
 Antibodies to Spinal Muscular Atrophy Gene Therapy Vector May Dissipate, Allowing More Children to Receive Treatment 1024 681 Katie Brind'Amour, PhD, MS, CHES
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sleeping infant

A review of antibody titers for SMA patients revealed that levels may decrease with time, potentially enabling delayed dosing for children who otherwise might have been excluded from life-saving gene therapy. Adeno-associated viral (AAV) vectors are used to deliver gene therapies such as onasemnogene abeparvovec-xioi (ZOLGENSMA®), a recently approved gene therapy for spinal muscular atrophy…

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Novel Genetic Variant Identified in CARMIL2 Deficiency
 Novel Genetic Variant Identified in CARMIL2 Deficiency 1024 575 Lauren Dembeck
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International program facilitates the diagnosis of a rare immunodeficiency in two children caused by a novel genetic variant. A multidisciplinary team of physicians and scientists at Nationwide Children’s Hospital have identified a novel loss-of-function variant causing CARMIL2 deficiency, an inborn error of immunity, in two Saudi Arabian brothers born to consanguineous parents. The case was…

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Mapping the World of Pediatric Severe Sepsis
 Mapping the World of Pediatric Severe Sepsis 1024 576 Katie Brind'Amour, PhD, MS, CHES
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Blood cells

Researchers work to reveal the many subtypes of pediatric sepsis — and what to do about them. Once upon a time, sepsis was just sepsis. Children experiencing septic shock and its aftermath — any resulting organ failure — were viewed as a fairly homogenous group of patients. But now, thanks in a large part to…

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Solving the Puzzle of Transfusion-Related Immune Reactions
 Solving the Puzzle of Transfusion-Related Immune Reactions 576 367 Katie Brind'Amour, PhD, MS, CHES
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Now that physicians have the “how” of blood transfusions mastered, they are starting to explore the “what” — and they’re learning that the effects of sharing human blood may be even more far-reaching and complex than previously imagined. With the initial safety challenges addressed and the technical barriers of donor blood storage and cleaning improved,…

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Harnessing the Immune System: Has the Cure for Cancer Been Within Us All Along?
 Harnessing the Immune System: Has the Cure for Cancer Been Within Us All Along? 1024 575 Abbie Miller
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By learning to manipulate the immune system to target cancer cells, clinician-scientists are ushering in a new era in cancer treatments. The advances in cancer immunotherapy have been headline-making, and some clinical studies have produced stories of near-miraculous recoveries. From the immunotherapy drug credited with curing former president Jimmy Carter’s cancer (pembrolizumab) to the promising…

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